Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party

Raffaella Greco, Attilio Bondanza, Maria Carolina Oliveira, Manuela Badoglio, Joachim Burman, Fredrik Piehl, Hans Hagglund, Eva Krasulova, Belinda Pinto Simões, Kristina Carlson, David Pohlreich, Myriam Labopin, Riccardo Saccardi, Giancarlo Comi, Gian Luigi Mancardi, Andrea Bacigalupo, Fabio Ciceri, Dominique Farge

Research output: Contribution to journalArticlepeer-review


Background: Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervoussystem, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse comparedwith multiple sclerosis.Objective: The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune DiseasesWorking Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologousstem cell transplantation (ASCT).Methods: This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patientssuffering from refractory NMO reported to the EBMT registry between 2001 and 2011.Results: Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one withG-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, afterconditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy(200 mg/kg) plus ATG (n = 4).After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while inthe remaining 13 patients further treatments were administered for disability progression or relapse afterASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, whileprogression-free survival remained 48% at three and five years.Conclusions: In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporarycontrol of the disease, despite a tendency to progress or relapse in the long term.

Original languageEnglish
Pages (from-to)189-197
Number of pages9
JournalMultiple Sclerosis Journal
Issue number2
Publication statusPublished - Feb 17 2015


  • Autologous stem cell transplantation
  • Disease outcome
  • Neuromyelitis optica

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology


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